Combined Effect of CFTR Protein Modulator Drugs and Exercise in Cystic Fibrosis

European University

Status: Withdrawn

Conditions: Cystic Fibrosis in Children

Treatments: Drug: CFTR Modulators; Behavioral: Exercise; Behavioral: Unsupervised exercise

Study type: Interventional

Funder types: Other

Identifiers

NCT04415268

CIPI/20/119

Details and patient eligibility

About

This study aims to assess the effects of programmed exercise combined with CFTR protein modulator drugs in the cardiorespiratory fitness, strength, functional capacity and agility in a group of young patients with Cystic Fibrosis.

Full description

The present study is a new clinical trial that extends our previous work intended to assess the effect of programmed exercise with or without electrical stimulation on cardiorespiratory fitness, strength, functional capacity and agility in a group of young patients with Cystic Fibrosis (ECOMIRIN) registered at ClinicalTrials.gov (Identifier: NCT04153669).

After finishing the study, a subset of the participants was treated with either TEZ/IVA or LUM/IVA per standard of care, as they were considered candidates according to the AEMPS (https://www.clinicaltrialsregister.eu/ctr-search/trial/2019-000833-37/ES). Those patients are expected to complete five months of pharmacological treatment without exercise by June 2020.

Primary Objective To evaluate the effect of an exercise program on physical fitness in cystic fibrosis patients after 5 months of administration of LUM/IVA or TEZ/IVA in comparison to the effect produced by the same exercise program before administration of the drug.

Secondary Objectives Other specific objectives include comparing the beneficial effects of exercise and LUM/IVA or TEZ/IVA on: 1) cardiorespiratory function and muscle strength; 2) the concentration of chlorine in sweat; and 3) quality of life.

Sex: All

Ages: 6 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• In order to be included, patients must have participated in the ECOMIRIN clinical trial (ClinicalTrials.gov Identifier: NCT04153669).
• They must have a previous CF diagnosis, defined as: sweat chlorine ≥ 60 mEq/L and/or carry two variants of the CFTR gene characterized as pathogenic.
• Age 6-18 years; eligible to receive treatment with TEZ/IVA or LUM/IVA according to the AEMPS (LUM/IVA for 6-11 year-old children homozygous for the F508del mutation; TEZ/IVA for ≥ 12 year-old children homozygous for the Phe508del mutation, as well as heterozygous for the F508 mutation plus one of the following mutations: P67L, R117C, L206W, R352Q, A455E, D579G, 711 + 3A G, S945L, S977F, R1070W, D1152H, 2789 + 5G A, 3272-26A G, and 3849 + 10kbC T);
• Agreement to collaborate in performing static and dynamic pulmonary function tests.
• Consent of children and/or parents or legal guardians to participate in the study after having read and understood the informed consent form.

Exclusion criteria

• CF patients not consenting to participate in the study at any time after reading and understanding the informed consent form will be excluded.
• CF patients who are not eligible to receive treatment with TEZ/IVA or LUM/IVA according to the AEMPS during the course of the study will be excluded.
• Study participants will be excluded if they test positive for any contagious viral infection that may affect the outcomes (e.g. SARS-CoV-2).

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Multitreatment

Experimental group

Description:

Pharmacological treatment per standard of care (whole study length, starting on week 1). Supervised exercise protocol (phase 1, 8 weeks starting on week 9). Unsupervised exercise protocol (phase 2, 8 weeks starting on week 17).

Treatment:

Behavioral: Unsupervised exercise

Drug: CFTR Modulators

Behavioral: Exercise